What is the mechanism of action of this treatment?

The most common treatments for Gaucher Disease include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). ERT works by supplementing the deficient enzyme, glucocerebrosidase, which helps break down the fatty substance glucocerebroside that accumulates in cells. This reduces the burden of the disease and alleviates symptoms. SRT, on the other hand, decreases the production of glucocerebroside, thereby reducing its accumulation. The investigational treatment LY3884961 is being studied for its potential to address peripheral manifestations of Gaucher Disease, likely by targeting similar pathways to improve enzyme function or reduce substrate accumulation. Understanding these mechanisms is crucial for Gaucher Disease patients as it directly impacts the effectiveness of the treatment in managing symptoms and improving quality of life.

What side effects are associated with this type of intervention?

Common treatments for Gaucher Disease include enzyme replacement therapy (ERT) with imiglucerase and substrate reduction therapy (SRT) with eliglustat. Imiglucerase is generally well-tolerated but can cause mild to moderate adverse events such as infusion reactions, headache, fever, and fatigue. Eliglustat, an oral SRT, has been associated with treatment-emergent adverse events in 92% of patients, including mild to moderate symptoms like headache, nausea, diarrhea, and fatigue. Both treatments aim to manage the symptoms and progression of Gaucher Disease effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Gaucher Disease, primarily focusing on enzyme replacement therapies (ERTs) and substrate reduction therapies (SRTs). ERTs such as imiglucerase (Cerezyme), velaglucerase alfa (VPRIV), and taliglucerase alfa (Elelyso) are designed to replace the deficient enzyme glucocerebrosidase. SRTs like miglustat (Zavesca) and eliglustat (Cerdelga) work by reducing the production of glucocerebroside, the substance that accumulates in Gaucher cells. These treatments aim to manage the symptoms and prevent the progression of the disease, similar to the goals of the investigational drug LY3884961 being studied for its safety and tolerability in patients with peripheral manifestations of Gaucher Disease.

What other types of research exist?

Promising novel alternatives to LY3884961 for Gaucher Disease patients include: 1) Venglustat, an oral substrate reduction therapy currently in clinical trials, 2) Eliglustat, another oral substrate reduction therapy that has shown efficacy in type 1 Gaucher Disease, and 3) Gene therapies like AVR-RD-02, which is in clinical development and aims to provide a long-term solution by addressing the genetic root of the disease.

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Monoclonal Antibodies

LY3884961 for Gaucher Disease

Phase 1 & 2

Recruiting

Research Sponsored by Prevail Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial tests a new drug called LY3884961 in adults with specific symptoms of Gaucher Disease. The study aims to find the safest and most effective dose by observing how patients' bodies react to different amounts of the drug. Researchers will monitor safety, side effects, and changes in disease symptoms over several years.

Who is the study for?

Adults aged 18-65 with Gaucher Disease (GD) and specific GBA1 mutations can join this trial. They must have been on enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years, but not responding well enough. Participants need to use effective birth control and cannot donate blood for a year.

What is being tested?

The study tests different doses of LY3884961 in adults with GD's peripheral symptoms. It's an open-label trial where everyone knows they're getting the drug, lasting about 5 years with close monitoring for safety, immune response, biomarkers, and effectiveness.

What are the potential side effects?

Since it's a dose-finding study for LY3884961, potential side effects are being evaluated; however typical concerns may include reactions at the injection site, liver issues due to medication metabolism or immune system responses affecting various organs.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence and severity of Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Secondary study objectives

Discontinuation of enzyme replacement therapy (ERT)/substrate reduction therapy (SRT)

GCase levels

GluSph levels

+3 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: LY3884961Experimental Treatment1 Intervention

LY3884961 is an advanced therapy investigational medicinal product administered as a single intravenous infusion.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Gaucher Disease include enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). ERT works by supplementing the deficient enzyme, glucocerebrosidase, which helps break down the fatty substance glucocerebroside that accumulates in cells. This reduces the burden of the disease and alleviates symptoms. SRT, on the other hand, decreases the production of glucocerebroside, thereby reducing its accumulation. The investigational treatment LY3884961 is being studied for its potential to address peripheral manifestations of Gaucher Disease, likely by targeting similar pathways to improve enzyme function or reduce substrate accumulation. Understanding these mechanisms is crucial for Gaucher Disease patients as it directly impacts the effectiveness of the treatment in managing symptoms and improving quality of life.